Updates

July 23, 2020August 3, 2020
My Butterfly Baby

In most cases I don’t agree that “ignorance is bliss.” In general, the more information we have, the better people we are and the ... Read more
July 22, 2020July 22, 2020
Report Describes Successful Treatment Combo Against Itching in EB Pruriginosa

The combination of the oral antidepressant mirtazapine with a topical gel containing ketamine and amitriptyline successfully treated excessive itching in a man with epidermolysis ... Read more
July 15, 2020July 15, 2020
Abeona Restarts VIITAL Phase 3 Trial of EB-101, Presents Positive Phase 1/2 Data

Abeona Therapeutics has restarted patient enrollment in the VIITAL Phase 3 clinical trial of EB-101, a potential gene-corrected cell therapy for people with recessive ... Read more
July 9, 2020July 9, 2020
National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, ... Read more
July 8, 2020July 8, 2020
IV Immunoglobulin May Help Treat Inflammation in Severe EBP, Case Report Suggests

Intravenous immunoglobulin (IVIG) may be a promising treatment for reducing inflammation in severe epidermolysis bullosa pruriginosa (EBP) — a rare type of dystrophic epidermolysis ... Read more

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January 23, 2018January 23, 2018

Factors Beyond COL7A1 Mutations May Contribute to Dystrophic EB Forms, Study Suggests

A study from Spain has raised the possibility that the development of dystrophic epidermolysis bullosa (DEB) could be attributable to additional factors besides mutations in the COL7A1 gene. Researchers reached this conclusion after studying three unrelated patients with different forms of ... Read more

Updates

My Butterfly Baby

Report Describes Successful Treatment Combo Against Itching in EB Pruriginosa

Abeona Restarts VIITAL Phase 3 Trial of EB-101, Presents Positive Phase 1/2 Data

IV Immunoglobulin May Help Treat Inflammation in Severe EBP, Case Report Suggests

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SD-101 Cream Lacks Wound Healing Superiority in Phase 3 Trial, Study Reports

2 New Mutations Linked to Epidermolysis Bullosa Pruriginosa in Case Study

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

CRISPR/Cas9 Gene Editing May Correct Genetic Defect in DEB, Study Suggests

Coalition Will Address Racial Disparities in Rare Disease Communities

Collaboration Seeks Treatments for Diseases Caused by Nonsense Mutations

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

PTR-01 Shows Signs of Efficacy in RDEB Patients, Early Trial Data Reveal

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Gentamicin Promotes Wound Closure in Severe JEB Patients, Phase 1/2 Trial Shows

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

EMA Names CRISPR-Cas9 Gene Editing Therapy an Orphan Drug for RDEB

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Phase 3 Trial of Gene Therapy B-VEC for DEB Expected to Start by June

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Cannabinoid Cream PPP004 Receives FDA Orphan Drug Designation to Treat EB

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Oral Dapsone Can Lead to Rapid, Sustained Improvement in Inherited EB, Case Report Shows

Early Study of RDEB Gene Therapy Using SkinGun as Delivery Vehicle

Phase 1 Trial of EB Cannabinol Cream Completes Treatment in Healthy Volunteers

NORD’s Advice on COVID-19: You’re Stronger Than You Think

$75M Funding to Advance Gene Therapy for RDEB Entering Phase 3 Trial

Factors Beyond COL7A1 Mutations May Contribute to Dystrophic EB Forms, Study Suggests

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My Butterfly Baby

Report Describes Successful Treatment Combo Against Itching in EB Pruriginosa

Abeona Restarts VIITAL Phase 3 Trial of EB-101, Presents Positive Phase 1/2 Data

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

IV Immunoglobulin May Help Treat Inflammation in Severe EBP, Case Report Suggests

Subscribe to our mailing list

SD-101 Cream Lacks Wound Healing Superiority in Phase 3 Trial, Study Reports

2 New Mutations Linked to Epidermolysis Bullosa Pruriginosa in Case Study

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

CRISPR/Cas9 Gene Editing May Correct Genetic Defect in DEB, Study Suggests

Coalition Will Address Racial Disparities in Rare Disease Communities

Collaboration Seeks Treatments for Diseases Caused by Nonsense Mutations

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

PTR-01 Shows Signs of Efficacy in RDEB Patients, Early Trial Data Reveal

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

Gentamicin Promotes Wound Closure in Severe JEB Patients, Phase 1/2 Trial Shows

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

EMA Names CRISPR-Cas9 Gene Editing Therapy an Orphan Drug for RDEB

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Phase 3 Trial of Gene Therapy B-VEC for DEB Expected to Start by June

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Cannabinoid Cream PPP004 Receives FDA Orphan Drug Designation to Treat EB

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Oral Dapsone Can Lead to Rapid, Sustained Improvement in Inherited EB, Case Report Shows

Early Study of RDEB Gene Therapy Using SkinGun as Delivery Vehicle

Phase 1 Trial of EB Cannabinol Cream Completes Treatment in Healthy Volunteers

NORD’s Advice on COVID-19: You’re Stronger Than You Think

$75M Funding to Advance Gene Therapy for RDEB Entering Phase 3 Trial

Pin It on Pinterest